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Showing Results for Duchenne muscular dystrophy
In the latest edition of touchREVIEWS in Neurology, we are pleased to present a collection of insightful articles that highlight the current landscape and future directions in neurological research and treatment. Firstly, Rajvinder Karda opens this issue with a compelling ...
Duchenne muscular dystrophy (DMD) is an X-linked recessive, progressive and universally fatal disease in the spectrum of dystrophinopathies,1Â with an incidence of 21.4 patients in 100,000 live male births worldwide.2,3 Historically, patients with DMD would lose ambulation by the age of 10 due ...
Duchenne muscular dystrophy (DMD) is caused by mutations in the dystrophin gene that disrupt the production of functional dystrophin protein, resulting in progressive muscle damage and loss of contractile function. Currently, multiple therapeutic agents that aim to restore dystrophin expression ...
Welcome to the spring edition of US Neurology. This journal aims to address topical subjects in the field of neurology to stimulate discussion focused on these issues. Articles have been chosen for their evaluation of current practices and research and ...
Welcome to the fall edition of US Neurology. This edition features a wide range of articles that provide an opportunity to review developments in the changing treatment landscape for neurological disorders and share expert opinions that should be of ...
Highlights Neuromuscular diseases exhibit a complex array of respiratory physiologic changes that typically progress over time. These physiologic changes can promote significant respiratory abnormalities during sleep, leading to decreased quality of life, significant daytime symptoms, and overall worse survival. ...
Duchenne muscular dystrophy (DMD) is a progressive, X-linked recessive neuromuscular disorder, characterized by muscle weakness, wasting, and degeneration starting in early childhood.1,2 DMD is caused by a mutation in the dystrophin gene that leads to an absence of functional dystrophin ...
Welcome to the latest edition of European Neurological Review, which features a diverse range of articles covering a number of therapeutic areas that directly affect neurologists and other practitioners involved in the care of patients with neurological illness. Our expert ...
These are exciting times in Duchenne muscular dystrophy (DMD); the first disease-modifying drug for this indication has been approved in the European Union for ambulatory DMD in patients >5 years; other drug treatments are in the pipeline.1–5 These treatments can ...
Duchenne muscular dystrophy (DMD), the most common and devastating type of muscular dystrophy,1 is characterised by the absence of the protein dystrophin, which causes premature muscle cell failure and leads to progressive muscle atrophy and loss. The condition is typically ...
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